Genetic Therapy Modes Quiz
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Questions and Answers

What is the main approach used in gene therapy for diseases caused by recessive mutations?

  • Complementing the defective gene (correct)
  • Introducing a normal gene
  • Targeting the protein product
  • Silencing the normal gene
  • What strategy is typically employed for autosomal dominant genetic diseases in gene therapy?

  • Introducing a normal gene
  • Activating the mutant protein
  • Silencing the defective gene (correct)
  • Increasing expression of the pathogenic gene
  • Which type of therapeutic RNA inhibits gene function by controlling gene function?

  • mRNAs
  • gRNAs
  • siRNAs (correct)
  • RNA aptamers
  • In gene therapy, what are RNAs that reprogram genetic information and include trans-splicing ribozymes used for?

    <p>Reprogramming genetic information</p> Signup and view all the answers

    What is the main characteristic of siRNAs used in RNA interference?

    <p>Double-stranded RNA with a 3′ overhang</p> Signup and view all the answers

    Which type of therapeutic RNA is used for DNA modification in gene therapy?

    <p>gRNAs</p> Signup and view all the answers

    What is the main function of Aptamers in cancer therapy?

    <p>Delivering chemotherapeutics to cancer cells</p> Signup and view all the answers

    How are SiRNAs generated by the ribonuclease?

    <p>Through endonucleolytic processing</p> Signup and view all the answers

    What is the main purpose of Ribozymes in gene therapy?

    <p>Downregulating pathogenic genes</p> Signup and view all the answers

    Which ribozyme motif is characterized by its ability for self-cleavage of a particular phosphodiester bond?

    <p>Hammerhead ribozyme</p> Signup and view all the answers

    What is the special characteristic of Group I and Group II intron ribozymes?

    <p>Self-splicing reaction</p> Signup and view all the answers

    Which disease has Ribozymes been clinically trialed for according to the text?

    <p>Solid tumors</p> Signup and view all the answers

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